Trial Outcomes & Findings for Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD) (NCT NCT02836418)
NCT ID: NCT02836418
Last Updated: 2023-12-22
Results Overview
TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. AEs were defined as any untoward medical occurrence in a participant administered study drug and that does not necessarily have a causal relationship with the study drug. Worsening of a pre-existing medical condition should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. SAEs were defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required in-participant hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in 'Reported Adverse Events' Section.
COMPLETED
PHASE1/PHASE2
8 participants
Up to End of Study (up to approximately Week 39)
2023-12-22
Participant Flow
Participants who participated in and completed the treatment period and were considered to be compliant with the study drug by the Investigator in the parent studies (ATYR1940-C-003 \[NCT02603562\] or ATYR1940-C-004 \[NCT02579239\]) were eligible for participation in this study. Note that only data for this study are reported in this Results Record. Data for the parent studies (ATYR1940-C-003 \[NCT02603562\] and ATYR1940-C-004 \[NCT02579239\]) are reported in the respective Results Records.
When a participant transferred from the parent study to this extension study, the duration between the last ATYR1940 dose in the parent study and first ATYR1940 dose in this extension study was to be 1 week; however, a maximum duration of 3 weeks was permissible.
Participant milestones
| Measure |
ATYR1940
Participants received ATYR1940 up to 3.0 milligrams per kilograms (mg/kg) intravenous (IV) infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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|---|---|
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Overall Study
STARTED
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8
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Overall Study
Received at Least One Dose of Study Drug
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8
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Overall Study
COMPLETED
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8
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
Baseline characteristics by cohort
| Measure |
ATYR1940
n=8 Participants
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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|---|---|
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Age, Continuous
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30.0 years
STANDARD_DEVIATION 15.94 • n=5 Participants
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Sex: Female, Male
Female
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1 Participants
n=5 Participants
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Sex: Female, Male
Male
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7 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Hispanic or Latino
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1 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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7 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Black or African American
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0 Participants
n=5 Participants
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Race (NIH/OMB)
White
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8 Participants
n=5 Participants
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Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Up to End of Study (up to approximately Week 39)Population: Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies.
TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. AEs were defined as any untoward medical occurrence in a participant administered study drug and that does not necessarily have a causal relationship with the study drug. Worsening of a pre-existing medical condition should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. SAEs were defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required in-participant hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in 'Reported Adverse Events' Section.
Outcome measures
| Measure |
ATYR1940
n=8 Participants
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
TEAEs
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7 Participants
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Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
SAEs
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0 Participants
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PRIMARY outcome
Timeframe: Up to End of Study (up to approximately Week 39)Population: Cumulative Safety population included all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies, but with cumulative data from both the parent studies and the current study.
Summarized titers are reported below.
Outcome measures
| Measure |
ATYR1940
n=8 Participants
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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Number of Participants With Positive Anti-Drug Antibodies (ADA)
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3 Participants
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PRIMARY outcome
Timeframe: Up to End of Study (up to approximately Week 39)Population: Cumulative Safety population included all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies, but with cumulative data from both the parent studies and the current study.
Participants with Jo-1 Ab levels ≥1.5 U/mL were to be discontinued from dosing of the study drug.
Outcome measures
| Measure |
ATYR1940
n=8 Participants
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL)
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2 Participants
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PRIMARY outcome
Timeframe: Up to End of Study (up to approximately Week 39)Population: Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies.
Laboratory parameters included hematology (hematocrit, hemoglobin, red blood cell count, white blood cell count neutrophils, lymphocytes, monocytes, eosinophils, basophils, and platelet count); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, alkaline phosphatase, total protein, sodium, potassium, bicarbonate, calcium, chloride, magnesium, inorganic phosphate, creatine kinase, lactate dehydrogenase, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, and cholesterol \[nonfasting\]); and urinalysis (color, pH, specific gravity, protein, glucose, ketones, and blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Outcome measures
| Measure |
ATYR1940
n=8 Participants
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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Number of Participants With a Clinical Laboratory Abnormality Leading to an AE
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1 Participants
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PRIMARY outcome
Timeframe: Up to End of Study (up to approximately Week 39)Population: Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies.
Pulmonary evaluations included pulmonary function tests and pulse oximetry. Clinically significant changes were to be reported as adverse events. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Outcome measures
| Measure |
ATYR1940
n=8 Participants
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE
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0 Participants
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PRIMARY outcome
Timeframe: Up to End of Study (up to approximately Week 39)Population: Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies.
ECG parameters that were evaluated included heart rate and PR, QR, and QT intervals. A clinically significant ECG abnormality was based upon the Investigator's discretion. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Outcome measures
| Measure |
ATYR1940
n=8 Participants
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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Number of Participants With a Clinically Significant Electrocardiogram (ECG) Abnormality Leading to a TEAE
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2 Participants
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PRIMARY outcome
Timeframe: Up to End of Study (up to approximately Week 39)Population: Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies.
The vital sign parameters that were evaluated included heart rate, systolic and diastolic blood pressure, and respiration rate as well as temperature. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Outcome measures
| Measure |
ATYR1940
n=8 Participants
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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Number of Participants With Vital Sign Abnormality Resulting in a TEAE
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0 Participants
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PRIMARY outcome
Timeframe: Baseline, Week 12Population: Cumulative Safety population included all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies, but with cumulative data from both the parent studies and the current study. Here, 'Number of Participants analyzed' signifies those who were evaluable for this outcome measure and number analyzed signifies those who were evaluable at specified time points only.
MMT (muscle strength) will be graded using a modified Medical Research Council scale. Scores were converted to 13-point scale (range from 0 \[on contraction palpable\] to 12 \[normal strength\]). An overall total score was calculated by summing all scores for a total possible score of 336. Decreased muscle strength was indicated by a decreased score. An overall total score was calculated as long as 24 of the 28 individual scores were non-missing.
Outcome measures
| Measure |
ATYR1940
n=7 Participants
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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Change From Baseline in Manual Muscle Testing (MMT) Score at Week 12
Baseline
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229.1 score on a scale
Standard Deviation 82.56
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Change From Baseline in Manual Muscle Testing (MMT) Score at Week 12
Change at Week 12
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4.3 score on a scale
Standard Deviation 13.50
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SECONDARY outcome
Timeframe: Baseline, Week 12Population: Cumulative Safety population included all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies, but with cumulative data from both the parent studies and the current study. Here, 'Number of Participants analyzed' signifies those who were evaluable for this outcome measure.
Outcome measures
| Measure |
ATYR1940
n=7 Participants
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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Change From Baseline in Creatinine Kinase at Week 12
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-82.3 Units/liter
Standard Deviation 164.98
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Adverse Events
ATYR1940
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
ATYR1940
n=8 participants at risk
Participants received ATYR1940 up to 3.0 mg/kg IV infusion once weekly until approval of ATYR1940, discontinuation of its development, the study was closed by the Sponsor, or a criterion for study drug discontinuation was met (up to a maximum of 34 weeks).
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Musculoskeletal and connective tissue disorders
Back pain
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37.5%
3/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Musculoskeletal and connective tissue disorders
Myalgia
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37.5%
3/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Musculoskeletal and connective tissue disorders
Pain in extremity
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37.5%
3/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Musculoskeletal and connective tissue disorders
Arthralgia
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Musculoskeletal and connective tissue disorders
Arthropathy
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Musculoskeletal and connective tissue disorders
Muscular weakness
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Musculoskeletal and connective tissue disorders
Musculoskeletal pain
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Gastrointestinal disorders
Nausea
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50.0%
4/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Gastrointestinal disorders
Diarrhoea
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Gastrointestinal disorders
Vomiting
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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General disorders
Fatigue
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25.0%
2/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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General disorders
Cyst
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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General disorders
Infusion site pain
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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General disorders
Oedema peripheral
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12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Injury, poisoning and procedural complications
Fall
|
50.0%
4/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Injury, poisoning and procedural complications
Back injury
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
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Injury, poisoning and procedural complications
Bone contusion
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Infections and infestations
Influenza
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Infections and infestations
Laryngitis
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Infections and infestations
Upper respiratory tract infection
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Investigations
Blood bicarbonate decreased
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Investigations
ECG signs of ventricular hypertrophy
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Investigations
Electrocardiogram P wave biphasic
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
25.0%
2/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Nervous system disorders
Headache
|
25.0%
2/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Nervous system disorders
Burning sensation
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Nervous system disorders
Tension headache
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
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Cardiac disorders
Atrioventricular block first degree
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Cardiac disorders
Right atrial dilatation
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Psychiatric disorders
Insomnia
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Reproductive system and breast disorders
Ovarian cyst
|
100.0%
1/1 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
|
|
Skin and subcutaneous tissue disorders
Rash
|
12.5%
1/8 • Baseline up to approximately Week 39
Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. Mortality, Serious Adverse Event, and Other Adverse Event data were prespecified to be collected as a single Arm/Group for any participant who received at least 1 dose of the study drug, regardless of their dose level.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place